Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) Via Direct Intravenous Injection of Lentiviral Vector (Ivlv-X1)
This is a Phase I/II gene therapy trial treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector (ivlv-X1) to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous lentiviral gene transfer protocol.
• Diagnosis of SCID-X1 based on:
‣ A proven mutation in the common interleukin-2 receptor gamma chain gene as defined by direct sequencing of patient DNA.
⁃ T-cell immune deficiency defined as one or more of the following: CD3+ autologous T cells \< 300/ul, or less than 50% of normal value for in vitro mitogen stimulation, or absent proliferation in vitro to antigen stimulation.
• No available HLA identical related donor.
• With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus; disseminated BCG infection.
• No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia.
• No prior allogeneic stem cell transplantation.
• Life expectancy ≥ 3 months.
• Documented to be negative for HIV infection.
• Written, informed consent obtained prior to any study-specific procedures.